TABLE 1.
Baseline Characteristics of Patients in Different Treatment Groups
| Characteristic | Randomized Controlled Trial | Open‐Label Trial | ||
|---|---|---|---|---|
| Preventive Treatment, n = 13 | Conventional Treatment, n = 14 | Preventive Treatment, n = 12 | Conventional Treatment, n = 15 | |
| Age at enrollment, days | ||||
| Mean ± SD | 36.1 ± 23.3 | 33.9 ± 27.6 | 40.2 ± 26.6 | 43.0 ± 26.6 |
| Median (IQR) | 36 (19–49) | 26.5 (16–35) | 33 (23–58) | 32 (19–68) |
| Sex, n (%) | ||||
| Female | 10 (77%) | 6 (43%) | 4 (33%) | 5 (33%) |
| Male | 3 (23%) | 8 (57%) | 8 (67%) | 10 (67%) |
| Mutation, n (%) | ||||
| TSC1 | 1 (8%) | 2 (14%) | 2 (17%) | 5 (33%) |
| TSC2 | 12 (92%) | 12 (86%) | 10 (83%) | 10 (67%) |
| No mutation identified | 0 (0%) | 0 (0%) | 0 (0%) | 0 (0%) |
| Brain MRI findings, n (%) | ||||
| Subependymal nodules | 12 (92%) | 14 (100%) | 12 (100%) | 13 (87%) |
| SEGAs | 1 (8%) | 1 (7%) | 2 (17%) | 0 (0%) |
| Radial migration lines | 10 (77%) | 12 (86%) | 10 (83%) | 10 (67%) |
| Tubers | 11 (85%) | 14 (100%) | 10 (83%) | 11 (73%) |
| Median tuber volume as proportion of total brain volume (range) | 2.896 (0.000–14.43) | 2.687 (0.115–11.039 | 2.813 (1.122–7.748) | 0.585 (0.000–6.669) |
Includes dropouts after treatment allocation and excludes patients who never had epileptiform activity/seizures and were not treated. No statistically significant differences between preventive and conventional groups and between randomized and open‐label trials were found.
IQR = interquartile range; MRI = magnetic resonance imaging; SD = standard deviation; SEGA = subependymal giant cell astrocytoma; TSC1 = tuberous sclerosis complex gene 1; TSC2 = tuberous sclerosis complex gene 2.